FDA accepts BLA for Bluebird’s lovo-cel for patients with SCD

bluebird bio announced that the U.S. Food and Drug Administration has accepted the Biologics License Application for lovotibeglogene autotemcel lovo-cel for priority review. Lovo-cel is a potentially transformative one-time gene therapy for individuals living with sickle cell disease ages 12 and older who have a history of vaso-occlusive events . It is specifically designed to treat the underlying cause of SCD through the addition of a functional gene that enables production of anti-sickling adult hemoglobin and is the most deeply studied gene therapy in development for this disease. The agency has set a Prescription Drug User Fee Act goal date of December 20. “The burden that people living with SCD and their families live with today is staggering. Beyond extreme pain crises that send patients to the hospital, SCD progression is associated with grave long-term consequences,” said Andrew Obenshain, chief executive officer, bluebird bio. “The FDA’s acceptance of our BLA for lovo-cel moves us one step closer in bringing a potentially transformative therapy to the sickle cell disease community that is long overdue, and we are grateful to the patients, caregivers, researchers, clinicians, and community leaders who have enabled this exciting milestone. We look forward to working with the agency on its review.”